IMPROVED THERAPEUTIC METHOD FOR RARE OCULAR DISEASES BY GENE REPLACEMENT

A recombinant adeno-associated virus (AAV) vector that carries a nucleic acid sequence encoding the retinal transcription factor cone-rod homeobox (CRX) for its use in treating CRX-associated IRDs in a subject in need thereof or for use in treating inherited retinal dystrophies caused by hypomorphic...

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Bibliographische Detailangaben
Hauptverfasser:	GRELLIER, Elodie Kim, ROGER, Jérôme
Format:	Patent
Sprache:	eng ; fre ; ger
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING ORGANIC CHEMISTRY PEPTIDES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	A recombinant adeno-associated virus (AAV) vector that carries a nucleic acid sequence encoding the retinal transcription factor cone-rod homeobox (CRX) for its use in treating CRX-associated IRDs in a subject in need thereof or for use in treating inherited retinal dystrophies caused by hypomorphic mutations in the CRX target genes.